Ravicti glycerol phenylbutyrate regulatory update

FDA approved an NDA for Ravicti glycerol phenylbutyrate from Hyperion for the management of chronic urea cycle disorders in patients >=2 years of age. The company said it plans to launch the drug in April, but did not disclose a price for the pre-prodrug of phenylacetic acid.

In January, FDA told the company to expect a delay on a decision for the NDA because the agency was "continuing to work on label and post-marketing requirements in connection" with the application, which had a Jan. 23 PDUFA date (see BioCentury, Jan. 21). Last September, FDA extended the PDUFA date by 3 months to Jan. 23 from Oct. 23, 2012. At the time, Hyperion said FDA needed more time to review additional data analyses submitted by the company at the agency's request (see BioCentury, Sept. 10, 2012). ...